trofinetide (Rx)

Brand and Other Names:Daybue

Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

oral solution

  • 200mg/mL (450mL/bottle)

Rett Syndrome

Indicated for treatment of Rett syndrome

35 kg to <50 kg: 10,000 mg (50 mL) PO BID

≥50 kg: 12,000 mg (60 mL) PO BID

Dosage Modifications

Diarrhea or weight loss

  • Advise patients to stop laxatives before initiating
  • Interrupt, reduce dosage, or discontinue if severe diarrhea occurs, if dehydration suspected, or if significant weight loss occurs

Renal impairment

  • No dedicated study has been conducted to evaluate pharmacokinetics in patients with renal impairment
  • Moderate-to-severe: Not recommended; eliminated mainly through the kidney

Hepatic impairment

  • No dose adjustment required; not significantly metabolized by liver

Dosing Considerations

Discontinue laxatives before initiating

Monitor weight during treatment

Dosage Forms & Strengths

oral solution

  • 200mg/mL (450mL/bottle)

Rett Syndrome

Indicated for treatment of Rett syndromein adults and children aged ≥2 years

9 kg to <12 kg: 5,000 mg (25 mL) PO BID

12 kg to <20 kg: 6,000 mg (30 mL) PO BID

20 kg to <35 kg: 8,000 (40 mL) PO BID

35 kg to <50 kg: 10,000 mg (50 mL) PO BID

≥50 kg: 12,000 mg (60 mL) PO BID

Dosage Modifications

Diarrhea or weight loss

  • Advise patients to stop laxatives before initiating
  • Interrupt, reduce dosage, or discontinue if severe diarrhea occurs, if dehydration suspected, or if significant weight loss occurs

Renal impairment

  • No dedicated study has been conducted to evaluate pharmacokinetics in patients with renal impairment
  • Moderate-to-severe: Not recommended; eliminated mainly through the kidney

Hepatic impairment

  • No dose adjustment required; not significantly metabolized by liver

Dosing Considerations

Discontinue laxatives before initiating

Monitor weight during treatment

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Interactions

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              Serious - Use Alternative (41)

              • ambrisentan

                trofinetide will increase the level or effect of ambrisentan by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • atogepant

                trofinetide will increase the level or effect of atogepant by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • atorvastatin

                trofinetide will increase the level or effect of atorvastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • bosentan

                trofinetide will increase the level or effect of bosentan by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • caspofungin

                trofinetide will increase the level or effect of caspofungin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • digoxin

                trofinetide will increase the level or effect of digoxin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • elagolix

                trofinetide will increase the level or effect of elagolix by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • elbasvir/grazoprevir

                trofinetide will increase the level or effect of elbasvir/grazoprevir by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • eluxadoline

                trofinetide will increase the level or effect of eluxadoline by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • enalapril

                trofinetide will increase the level or effect of enalapril by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • erythromycin base

                trofinetide will increase the level or effect of erythromycin base by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • erythromycin ethylsuccinate

                trofinetide will increase the level or effect of erythromycin ethylsuccinate by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • erythromycin lactobionate

                trofinetide will increase the level or effect of erythromycin lactobionate by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • erythromycin stearate

                trofinetide will increase the level or effect of erythromycin stearate by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • ezetimibe

                trofinetide will increase the level or effect of ezetimibe by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • fexofenadine

                trofinetide will increase the level or effect of fexofenadine by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • fluvastatin

                trofinetide will increase the level or effect of fluvastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • glecaprevir/pibrentasvir

                trofinetide will increase the level or effect of glecaprevir/pibrentasvir by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • glyburide

                trofinetide will increase the level or effect of glyburide by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • imatinib

                trofinetide will increase the level or effect of imatinib by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • letermovir

                trofinetide will increase the level or effect of letermovir by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • levothyroxine

                trofinetide will increase the level or effect of levothyroxine by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • lovastatin

                trofinetide will increase the level or effect of lovastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • methotrexate

                trofinetide will increase the level or effect of methotrexate by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • olmesartan

                trofinetide will increase the level or effect of olmesartan by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • paclitaxel

                trofinetide will increase the level or effect of paclitaxel by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • penicillin G benzathine

                trofinetide will increase the level or effect of penicillin G benzathine by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • pitavastatin

                trofinetide will increase the level or effect of pitavastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • pravastatin

                trofinetide will increase the level or effect of pravastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • repaglinide

                trofinetide will increase the level or effect of repaglinide by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • revefenacin

                trofinetide will increase the level or effect of revefenacin by Other (see comment). Avoid or Use Alternate Drug. rofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • rifampin

                trofinetide will increase the level or effect of rifampin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • rosuvastatin

                trofinetide will increase the level or effect of rosuvastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • sacubitril/valsartan

                trofinetide will increase the level or effect of sacubitril/valsartan by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • simvastatin

                trofinetide will increase the level or effect of simvastatin by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • sulbactam/durlobactam

                trofinetide will increase the level or effect of sulbactam/durlobactam by Other (see comment). Avoid or Use Alternate Drug. Sulbactam is predicted to have active secretion by OATP1 as a significant portion of total clearance; therefore, inhibition of OAT1 may increase sulbactam plasma concentrations

              • telmisartan

                trofinetide will increase the level or effect of telmisartan by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • troglitazone

                trofinetide will increase the level or effect of troglitazone by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • valsartan

                trofinetide will increase the level or effect of valsartan by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • voxilaprevir

                trofinetide will increase the level or effect of voxilaprevir by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              • zavegepant intranasal

                trofinetide will increase the level or effect of zavegepant intranasal by Other (see comment). Avoid or Use Alternate Drug. Trofinetide (an OATP131 and OATP13B inhibitor) may increase plasma levels of OATP131 or OATP13B substrates. Avoid coadministration with sensitive substrates.

              Monitor Closely (30)

              • alfentanil

                trofinetide will increase the level or effect of alfentanil by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • carbamazepine

                trofinetide will increase the level or effect of carbamazepine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • clonidine

                trofinetide will increase the level or effect of clonidine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • colchicine

                trofinetide will increase the level or effect of colchicine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • cyclosporine

                trofinetide will increase the level or effect of cyclosporine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • dihydroergotamine

                trofinetide will increase the level or effect of dihydroergotamine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • disopyramide

                trofinetide will increase the level or effect of disopyramide by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • divalproex sodium

                trofinetide will increase the level or effect of divalproex sodium by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • ergotamine

                trofinetide will increase the level or effect of ergotamine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • ethosuximide

                trofinetide will increase the level or effect of ethosuximide by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • everolimus

                trofinetide will increase the level or effect of everolimus by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • fentanyl

                trofinetide will increase the level or effect of fentanyl by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • fosphenytoin

                trofinetide will increase the level or effect of fosphenytoin by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • midazolam

                trofinetide will increase the level or effect of midazolam by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • momelotinib

                trofinetide increases toxicity of momelotinib by Other (see comment). Use Caution/Monitor. Comment: OATP1B1/B3 inhibitor increases momelotinib (OATP1B1/B3 subtrate) plasma concentrations, which may increase the risk of adverse reactions with momelotinib.

              • pacritinib

                trofinetide will increase the level or effect of pacritinib by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • phenobarbital

                trofinetide will increase the level or effect of phenobarbital by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • phenytoin

                trofinetide will increase the level or effect of phenytoin by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • pimozide

                trofinetide will increase the level or effect of pimozide by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • primidone

                trofinetide will increase the level or effect of primidone by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • quinidine

                trofinetide will increase the level or effect of quinidine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • quinine

                trofinetide will increase the level or effect of quinine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • repaglinide

                trofinetide will increase the level or effect of repaglinide by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • sirolimus

                trofinetide will increase the level or effect of sirolimus by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • tacrolimus

                trofinetide will increase the level or effect of tacrolimus by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • theophylline

                trofinetide will increase the level or effect of theophylline by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • thioridazine

                trofinetide will increase the level or effect of thioridazine by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • triazolam

                trofinetide will increase the level or effect of triazolam by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • valproic acid

                trofinetide will increase the level or effect of valproic acid by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              • warfarin

                trofinetide will increase the level or effect of warfarin by affecting hepatic/intestinal enzyme CYP3A4 metabolism. Use Caution/Monitor. Monitor CYP3A4 substrates for which a small increase in plasma concentration may lead to serious toxicities if coadministered with trofinetide (a weak CYP3A4 inhibitor).

              Minor (0)

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                Adverse Effects

                >10%

                Diarrhea (82%)

                Diarrhea, persistent or recurrent after dose interruptions, reductions, or concomitant antidiarrheal therapy (49%)

                Vomiting (29%)

                1-10%

                Fever (9%)

                Seizure (9%)

                Anxiety (8%)

                Decreased appetite (8%)

                Fatigue (8%)

                Nasopharyngitis (5%)

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                Warnings

                Contraindications

                None

                Cautions

                Diarrhea

                • Diarrhea expected in most patients and ranges in severity
                • Advise patients to stop laxatives before starting
                • If diarrhea occurs, have patient notify healthcare provider
                • Consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed
                • Interrupt, reduce dose, or discontinue if severe diarrhea occurs or if dehydration suspected

                Weight loss

                • Weight loss may occur
                • Monitor weight and interrupt, reduce dose, or discontinue if significant weight loss occurs

                Drug interaction overview

                Inhibitor of CYP3A4 (weak), OATP1B1, and OATP1B3

                • Sensitive CYP3A4 substrates
                  • Closely monitor
                  • Coadministration may increase plasma concentrations of orally administered CYP3A4 sensitive substrates
                  • Monitor CYP3A4 substrates for which a small change in substrate plasma concentration may lead to serious toxicities
                • Sensitive OATP1B1 or OATP13 substrates
                  • Avoid coadministration
                  • Trofinetide inhibits OATP1B1 and OATP1B3 in vitro
                  • Avoid with substrates for which a small change in substrate plasma concentration may lead to serious toxicities
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                Pregnancy & Lactation

                Pregnancy

                There are no adequate data on developmental risks associated with use in pregnant females

                Animal studies

                • No adverse developmental effects observed following administration to pregnant animals at doses associated with plasma exposures below those used clinically

                Lactation

                Data are not available regarding presence of trofinetide or its metabolites in human milk, effects on breastfed infants, or effects on milk production

                Pregnancy Categories

                A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

                B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

                C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

                D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

                X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

                NA: Information not available.

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                Pharmacology

                Mechanism of Action

                Mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown

                Synthetic analog of the amino‐terminal tripeptide of insulinlike growth factor (IGF-1) designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function, specifically, the protein fragment glypromate (GPE)

                GPE consist of the amino acids glycine-proline-glutamate; trofinetide is a modified form of GPE allowing it to be stable and administered orally, unlike GPE or IGF-1

                IGF-1 is produced by both major types of brain cells – neurons and glia; IGF-1 in the brain is critical for both normal development and response to injury and disease

                Trofinetide has been shown to inhibit the production of inflammatory cytokines, inhibit overactivation of microglia and astrocytes, and increase amount of available IGF-1 that can bind to IGF-1 receptors

                Absorption

                Peak plasma time: 2-3 hr

                Bioavailability: ≥84%

                Effect of food: High-fat meal had not impact on AUC and reduce peak plasma concentration ~20%

                Distribution

                Protein bound: <6%

                Vd: ~80 L

                Metabolism

                Not significantly metabolized by CYP450 enzymes

                Hepatic metabolism is not a significant route of elimination

                Elimination

                Half-life: 1.5 hr

                Excretion: Urine ~80% (unchanged); feces (minor)

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                Administration

                Oral Administration

                May take with or without food

                Administer orally or via gastrostomy (G) tube; doses administered via gastrojejunal (GJ) tubes must be administered through the G-port

                Use calibrated measuring device (eg, oral syringe or oral dosing cup) to measure and deliver prescribed dose accurately; household measuring cup is not an adequate measuring device

                Missed or vomited dose

                • If dose missed, take next dose as scheduled; do not double dose
                • If dose vomited, do not take an additional dose; instead, continue with next scheduled dose

                Storage

                • Refrigerate bottle in upright position at 2-8ºC (36-46ºF)
                • Do not freeze
                • Keep child-resistant cap tightly closed
                • Discard any unused oral solution after 14 days of first opening bottle
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                Images

                No images available for this drug.
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                Patient Handout

                A Patient Handout is not currently available for this monograph.
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                Formulary

                FormularyPatient Discounts

                Adding plans allows you to compare formulary status to other drugs in the same class.

                To view formulary information first create a list of plans. Your list will be saved and can be edited at any time.

                Adding plans allows you to:

                • View the formulary and any restrictions for each plan.
                • Manage and view all your plans together – even plans in different states.
                • Compare formulary status to other drugs in the same class.
                • Access your plan list on any device – mobile or desktop.

                The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

                Tier Description
                1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
                2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
                3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
                4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                NC NOT COVERED – Drugs that are not covered by the plan.
                Code Definition
                PA Prior Authorization
                Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
                QL Quantity Limits
                Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
                ST Step Therapy
                Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
                OR Other Restrictions
                Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
                Additional Offers
                Email to Patient

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                Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.